Department of Molecular Medicine, Central European Institute of Technology, Masaryk University (CEITEC MU), Kamenice 5, 62500, Brno, Czech Republic,
Adv Exp Med Biol. 2013;792:109-31. doi: 10.1007/978-1-4614-8051-8_5.
CLL patients harboring TP53 defects remain the most challenging group in terms of designing rational and effective therapy. Irrespective of the treatment employed-chemotherapy, chemoimmunotherapy, or pure biological drugs-median survival of these patients does not exceed 3-4 years. This adverse outcome is caused by a less effective response to therapeutics acting through DNA damage induction and relying on the subsequent initiation of apoptosis as well as by virtually inevitable aggressive relapse. Patient proportions with TP53 defects at diagnosis or before first therapy were reported within the range 5-15 %, but they increase dramatically in pretreated cohorts (reported up to 44 %), and also in patients with Richter transformation (50 % harbor TP53 defects). Currently, most laboratories monitor TP53 defect as presence of 17p deletion using I-FISH, but 23-45 % of TP53-affected patients were shown to harbor only mutation(s). In other patients with intact TP53, the p53 pathway may be impaired by mutations in ATM gene coding for the p53-regulatory kinase; however, prognosis of ATM-defective patients is not as poor as those with TP53 abnormalities. Though many novel agents are under development, the monoclonal antibody alemtuzumab and allogeneic stem cell transplantation remain the basic treatment options for TP53-affected CLL patients.
在设计合理有效的治疗方案方面,携带 TP53 缺陷的 CLL 患者仍然是最具挑战性的群体。无论采用何种治疗方法——化疗、化疗免疫治疗还是单纯的生物药物治疗——这些患者的中位生存时间都不超过 3-4 年。这种不良预后是由于对通过诱导 DNA 损伤并依赖随后启动细胞凋亡起作用的治疗方法的反应效果较差所致,也是由于几乎不可避免的侵袭性复发所致。在诊断或首次治疗前,有报道称患者中携带 TP53 缺陷的比例为 5-15%,但在预处理队列中显著增加(报道高达 44%),在 Richter 转化的患者中也显著增加(50%携带 TP53 缺陷)。目前,大多数实验室通过 I-FISH 监测 TP53 缺陷作为 17p 缺失的存在,但据报道,23-45%的 TP53 相关患者仅携带突变(s)。在其他具有完整 TP53 的患者中,p53 调节激酶 ATM 基因的突变可能会损害 p53 途径;然而,ATM 缺陷患者的预后并不像那些具有 TP53 异常的患者那样差。尽管正在开发许多新型药物,但单克隆抗体阿仑单抗和异基因干细胞移植仍然是 TP53 相关 CLL 患者的基本治疗选择。